Approval of the gene therapy will allow treatment for people with vision-loss due to RPE65 mutations and will help advance other investigational studies with potential to help millions, FFB tells the Committee

As noted by the earlier FDA decision to give voretigene neparvovec a priority review, the treatment will be groundbreaking for patients with vision loss due to a mutation in the RPE65 gene. If approved, voretigene neparvovec has the potential to be the first FDA approved gene therapy for the eye and for any inherited disease.

“The approval of this gene therapy will be life-changing for people with severe vision loss due to RPE65 mutations,” Dr. Stephen Rose, FFB’s Chief Research Officer, told the committee during its hearing. “FDA approval of this groundbreaking treatment will provide strong momentum for the advancement of several other vision-saving gene therapies under development in labs and clinics around the world”.

The investigational treatment, the result of more than two decades of research and development, delivers functional copies of the RPE65 gene directly into the retina thereby compensating for nonfunctional, mutated copies. FFB was an early financial supporter of that work investing $10 million for RPE65 lab and clinical research. FFB applauds the investigative teams at the University of Pennsylvania, Children’s Hospital of Philadelphia (CHOP) and at Spark Therapeutics for bringing the therapy into and through clinical trials that have demonstrated safety and strong efficacy.

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The Foundation Fighting Blindness was established in 1971. It has since raised more than $700 million toward its mission to prevent, treat and cure blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, experience vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.